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2025 Annual Meeting | S31 - Hot Topics in Child Neurology

Tuesday 04/08/25
03:30 PM - 05:30 PM PDT Add To Calendar
San Diego Convention Center | 28D
This program will be presented both in-person and online
This program will be available in the meeting's On Demand product.
Scientific Platform Session
Duygu Selcen, MD, FAAN, Courtney Wusthoff, MD
Child Neurology and Developmental Neurology
2.00 CME credits

Program Materials Program Evaluations

Event Timeline
03:30 PM - 03:42 PM PDT Abstract 001 - Treatment with UX111 Gene Therapy Rapidly Reduced Heparan Sulfate (HS) Exposure in Cerebrospinal Fluid (CSF) and Improved Long-term Cognitive Function in Children with Mucopolysaccharidosis IIIA (MPS IIIA)
Heather Lau, MD
03:42 PM - 03:54 PM PDT Abstract 002 - Large Cohort Integrative Multiomic Analyses Identifies Novel Risk Genes for Congenital Cerebral Ventriculomegaly and Characterizes Distinct Spatiotemporal Clusters of Pathogenesis
Garrett Allington
03:54 PM - 04:06 PM PDT Abstract 003 - Children with Peri-Optic Neuritis Have Favorable Visual Outcomes After Steroid Treatment: A Singe-Center Pediatric Case Series
Nicholas Benjamin, MD
04:06 PM - 04:18 PM PDT Abstract 004 - Subgroup and Subdomain Analysis of Patients with Long Term Follow-Up In a Phase III, Randomized, Placebo-Controlled Crossover Trial with Levacetylleucine (IB1001) for Niemann-Pick Disease Type C
Marc C. Patterson, MD, FRACP, FAAN
04:18 PM - 04:30 PM PDT Abstract 005 - Folic Acid in Pregnancy and Neuropsychological Outcomes at Age 6-Years-Old in the MONEAD Study
Kimford J. Meador, MD, FAAN
04:30 PM - 04:42 PM PDT Abstract 006 - Autism Spectrum Disorder Detection Using Resting-State fMRI Imaging with Convolutional Neural Networks
Xiao Chang, PhD
04:42 PM - 04:54 PM PDT Abstract 007 - Long-term Safety and Effectiveness of ZYN002 Cannabidiol Transdermal Gel in the Treatment of Irritability-Related Behavioral Symptoms in Children and Adolescents with Fragile X Syndrome: Update to Open-Label Extension Study (ZYN2-CL-017)
Kristen Bzdek, MD
04:54 PM - 05:06 PM PDT Abstract 008 - Lesion Network Mapping of Domains of Intellectual Functioning in Perinatal Strokes
Shreya Tripathy
05:06 PM - 05:18 PM PDT Abstract 009 - Serial Compound Muscle Action Potential Recordings in Duchenne Muscular Dystrophy
Mary K. Vollmer
05:18 PM - 05:30 PM PDT Abstract 010 - Safety and Efficacy of DYNE-251 in Males with DMD Mutations Amenable to Exon 51 Skipping
Perry Shieh, MD, PhD, FAAN
Faculty Disclosures
Shreya Tripathy Ms. Tripathy has nothing to disclose.
Clara Steeby Ms. Steeby has nothing to disclose.
Gillian Miller Ms. Miller has nothing to disclose.
Alexander L. Cohen, MD, PhD, FAAN The institution of Dr. Cohen has received research support from NIH. The institution of Dr. Cohen has received research support from Simons Foundation.
Laura L. Lehman, MD The institution of Dr. Lehman has received research support from Children's Tumor Foundation . Dr. Lehman has received personal compensation in the range of $500-$4,999 for serving as a Consultant with Clinical Neurology Society of America.
Xiao Chang, PhD Dr. Chang has received research support from National Science Foundation.
Md Mahmudul Hasan Mr. Hasan has nothing to disclose.
Gopikrishna Deshpande, PhD Dr. Deshpande has nothing to disclose.
Kimford J. Meador, MD, FAAN The institution of Dr. Meador has received research support from NIH. The institution of Dr. Meador has received research support from Eisai. The institution of Dr. Meador has received research support from Medtronics. The institution of Dr. Meador has received research support from The Epilepsy Consortium.
Morris Cohen Morris Cohen has received research support from Stanford University. Morris Cohen has received publishing royalties from a publication relating to health care.
David W. Loring, PhD, FAAN Dr. Loring has received personal compensation in the range of $5,000-$9,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Springer Nature. Dr. Loring has received personal compensation in the range of $5,000-$9,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for ILAE. The institution of Dr. Loring has received research support from NIH. Dr. Loring has received publishing royalties from a publication relating to health care.
Abigail Matthews No disclosure on file
Page B. Pennell, MD, FAAN The institution of Dr. Pennell has received research support from NIH. Dr. Pennell has received publishing royalties from a publication relating to health care.
Garrett Allington Mr. Allington has nothing to disclose.
Kristopher Kahle No disclosure on file
Kedous Y. Mekbib, MD Dr. Mekbib has nothing to disclose.
Evan Dennis Mr. Dennis has nothing to disclose.
Danielle F. Miyagishima, PhD The institution of Dr. Miyagishima has received research support from NIH-NCI.
Janelle Raymond, PhD Dr. Schafer has received personal compensation for serving as an employee of IntraBio. Dr. Schafer has stock in Amylyx Pharmaceuticals .
Bethany Zanrucha Bethany Zanrucha has stock in IntraBio. Bethany Zanrucha has stock in Sarepta.
Michael Strupp, MD, DO, FAAN Dr. Strupp has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Vertify. Dr. Strupp has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for IntraBio. Dr. Strupp has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Vifor, Frisenius, CH. Dr. Strupp has received personal compensation in the range of $10,000-$49,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Springer. Dr. Strupp has received personal compensation in the range of $5,000-$9,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Frontiers. Dr. Strupp has stock in IntraBio.
Tatiana T. Bremova-Ertl, MD, PhD Dr. Bremova-Ertl has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Intrabio. The institution of Dr. Bremova-Ertl has received research support from InnoSuisse. Dr. Bremova-Ertl has received research support from Baasch-Medicus.
Ian Billington, PhD Mr. Billington has received personal compensation for serving as an employee of IntraBio Inc. Mr. Billington has stock in IntraBio Inc.
Kristen Bzdek, MD Dr. Bzdek has received personal compensation for serving as an employee of Harmony Biosciences. Dr. Bzdek has stock in Harmony Biosciences.
Anthony Thibodeau Mr. Thibodeau has received personal compensation for serving as an employee of Harmony Biosciences.
Nancy R. Tich, PhD Dr. Tich has received personal compensation for serving as an employee of Harmony Biosciences. Dr. Tich has stock in Harmony Biosciences.
David Albers, PhD Dr. Albers has received personal compensation for serving as an employee of Harmony Biosciences. Dr. Albers has stock in Harmony Biosciences.
George G. Nomikos, MD, PhD Dr. Nomikos has received personal compensation for serving as an employee of Harmony Biosciences.
Mary K. Vollmer Ms. Vollmer has nothing to disclose.
Sumit Verma, MD Dr. Verma has nothing to disclose.
Perry Shieh, MD, PhD, FAAN Dr. Shieh has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sarepta. Dr. Shieh has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Genentech. Dr. Shieh has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Biogen. Dr. Shieh has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion. Dr. Shieh has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Argenx. Dr. Shieh has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for UCB. Dr. Shieh has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for CSL Behring. Dr. Shieh has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Grifols. Dr. Shieh has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Alexion. Dr. Shieh has received personal compensation in the range of $50,000-$99,999 for serving on a Speakers Bureau for Biogen. Dr. Shieh has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Genentech. Dr. Shieh has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Argenx. Dr. Shieh has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Catalyst.
Craig G. Campbell, MD Dr. Campbell has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Catabasis. Dr. Campbell has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Solid. The institution of Dr. Campbell has received research support from Biogen. The institution of Dr. Campbell has received research support from Genzyme. The institution of Dr. Campbell has received research support from Acceleron, AMO, Biogen, Sarepta, Wave, PTC, Pfizer, Roche.
Nicholas Deconinck Nicholas Deconinck has received personal compensation in the range of $500-$4,999 for serving as a Consultant for novartis. Nicholas Deconinck has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for roche. Nicholas Deconinck has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for biogen. Nicholas Deconinck has received publishing royalties from a publication relating to health care.
Liesbeth De Waele, MD, PhD Dr. De Waele has nothing to disclose.
Kevin M. Flanigan, MD, FAAN Dr. Flanigan has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sarepta. Dr. Flanigan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Apic Bio. Dr. Flanigan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for AavantiBio. Dr. Flanigan has stock in 4D Molecular Therapeutics. The institution of Dr. Flanigan has received research support from Abeona Therapeutics. The institution of Dr. Flanigan has received research support from Sarepta Therapeutics. The institution of Dr. Flanigan has received research support from Astellas Therapeutics. Dr. Flanigan has received intellectual property interests from a discovery or technology relating to health care.
Georgie Lorentzos No disclosure on file
Han Phan, MD Dr. Phan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Wave Biosciences. Dr. Phan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Stealth.
Chris Mix, MD Dr. Mix has received personal compensation for serving as an employee of Dyne Therapeutics. Dr. Mix has stock in Dyne Therapeutics.
Soma S. Ray Soma S. Ray has received personal compensation for serving as an employee of Dyne Therapeutics. Soma S. Ray has stock in Dyne Therapeutics.
Ashish Dugar No disclosure on file
Dazhe Wang, PhD Dr. Wang has received personal compensation for serving as an employee of Dyne Therapeutics. Dr. Wang has stock in Dyne Therapeutics.
Douglas A. Kerr, MD, PhD Dr. Kerr has received personal compensation for serving as an employee of Dyne Therapeutics. Dr. Kerr has received personal compensation for serving as an employee of Generation Bio. Dr. Kerr has received personal compensation for serving as an employee of Atlas Venture. Dr. Kerr has received personal compensation in the range of $10,000-$49,999 for serving as an officer or member of the Board of Directors for BlueRock Therapeutics. Dr. Kerr has received personal compensation in the range of $10,000-$49,999 for serving as an officer or member of the Board of Directors for AAVantgarde Therrapeutics. Dr. Kerr has received personal compensation in the range of $10,000-$49,999 for serving as an officer or member of the Board of Directors for Trace Neuroscience. Dr. Kerr has stock in Dyne Therapeutics. Dr. Kerr has stock in Generation Bio.
Maria Naylor, PhD Dr. Naylor has received personal compensation for serving as an employee of Dyne Therapeutics. Dr. Naylor has stock in Biogen. Dr. Naylor has stock in Dyne Therapeutics.
Michela Guglieri The institution of Michela Guglieri has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pfizer. The institution of Michela Guglieri has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Dyne. The institution of Michela Guglieri has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for NS Pharma. Michela Guglieri has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Sarepta. The institution of Michela Guglieri has received research support from EC - H2020. The institution of Michela Guglieri has received research support from Duchenne UK. The institution of Michela Guglieri has received research support from Sarepta. The institution of Michela Guglieri has received research support from DMD Research Fund.
Nicholas Benjamin, MD Dr. Benjamin has nothing to disclose.
Levi C. Shelton, MD Dr. Shelton has nothing to disclose.
Catalina Cleves, MD Dr. Cleves has nothing to disclose.
Srikala Narayanan, MD An immediate family member of Dr. Narayanan has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Vertex Pharmaceuticals. An immediate family member of Dr. Narayanan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Emerging Therapies Solutions. The institution of an immediate family member of Dr. Narayanan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Jazz Pharmaceuticals. The institution of an immediate family member of Dr. Narayanan has received research support from Jazz Pharmaceuticals.
Heather Lau, MD Dr. Lau has received personal compensation for serving as an employee of Ultragenyx. Dr. Lau has stock in Ultragenyx.
Kaushik Patra, PhD Dr. Patra has received personal compensation for serving as an employee of Ultragenyx. Dr. Patra has stock in Ultragenyx.
Melissa Wolf, PT Mrs. Wolf has received personal compensation for serving as an employee of Ultragenyx Pharmaceutical. Mrs. Wolf has stock in Ultragenyx Pharmaceutical.
Nicholas Smith, MBBS, PhD, FRACP The institution of Dr. Smith has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Forge Bio. The institution of Dr. Smith has received research support from Ultragenyx. The institution of Dr. Smith has received research support from Cyclotherapeutics. The institution of Dr. Smith has received research support from BluebirdBio. The institution of Dr. Smith has received research support from Sanfilippo Children's Foundation. The institution of Dr. Smith has received research support from Medical Research Future Fund. The institution of Dr. Smith has received research support from Women's and Children's Hospital Foundation.
María Luz Couce, MD, PhD Prof. Couce has nothing to disclose.
Deepa S. Rajan, MD, FAAN Dr. Rajan has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Takeda. Dr. Rajan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. Dr. Rajan has received personal compensation in the range of $0-$499 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Medlink Neurology. The institution of Dr. Rajan has received research support from National Scleroderma Foundation. The institution of Dr. Rajan has received research support from Denali Therapeutics. The institution of Dr. Rajan has received research support from Ultragenyx . The institution of Dr. Rajan has received research support from Regenxbio. The institution of Dr. Rajan has received research support from Takeda. The institution of Dr. Rajan has received research support from Prevail Therapeutics. The institution of Dr. Rajan has received research support from Children's Neuroscience Institute. Dr. Rajan has received intellectual property interests from a discovery or technology relating to health care.
Kristen Truxal, MD The institution of Dr. Truxal has received research support from Ultragenyx. The institution of Dr. Truxal has received research support from Inozyme Therapeutics.
María Jose Lopez, MD, PhD Dr. Lopez has nothing to disclose.
Maria Fuller, PhD The institution of Prof. Fuller has received research support from Alexion. The institution of Prof. Fuller has received research support from Ultragenyx. The institution of Prof. Fuller has received research support from Sanofi. The institution of Prof. Fuller has received research support from Paradigm.
Eines Monteagudo, MD Mrs. Monteagudo has nothing to disclose.
Lucy Dougherty, MD Ms. Dougherty has nothing to disclose.
Mireia del Toro, MD Dr. del Toro has nothing to disclose.
Duygu Selcen, MD, FAAN Dr. Selcen has nothing to disclose.
Marc C. Patterson, MD, FRACP, FAAN Dr. Patterson has received personal compensation for serving as an employee of IntraBio. The institution of Dr. Patterson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Orphazyme /KemPharm/Zevra. Dr. Patterson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Azafaros. Dr. Patterson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for IntraBio. Dr. Patterson has received personal compensation in the range of $10,000-$49,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Sage. Dr. Patterson has stock in IntraBio. The institution of Dr. Patterson has received research support from Glycomine. The institution of Dr. Patterson has received research support from Orphazyme /KemPharm/Zevra. The institution of Dr. Patterson has received research support from Idorsia. The institution of Dr. Patterson has received research support from Shire-Takeda. Dr. Patterson has received publishing royalties from a publication relating to health care. Dr. Patterson has received publishing royalties from a publication relating to health care.
Courtney Wusthoff, MD Dr. Wusthoff has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for ICON. Dr. Wusthoff has received personal compensation in the range of $10,000-$49,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Neurology. The institution of Dr. Wusthoff has received research support from NIH. Dr. Wusthoff has received publishing royalties from a publication relating to health care.